2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Beemer-Langer syndrome | antisense oligonucleotide consisting of 18 nucleotide residues with a sequence of 5¿-5MeC-5MeU-5MeU-5MeU-5MeC-5MeC-5MeU-5MeU-5MeC-A-5MeC-5MeU-G-5MeU- 5MeU-G-5MeC-A-3¿ | Orphan Designation | - |
| brain-lung-thyroid syndrome | antisense oligonucleotide consisting of 18 nucleotide residues with a sequence of 5¿-5MeC-5MeU-5MeU-5MeU-5MeC-5MeC-5MeU-5MeU-5MeC-A-5MeC-5MeU-G-5MeU- 5MeU-G-5MeC-A-3¿ | Orphan Designation | - |
| cranioectodermal dysplasia 2 | antisense oligonucleotide consisting of 18 nucleotide residues with a sequence of 5¿-5MeC-5MeU-5MeU-5MeU-5MeC-5MeC-5MeU-5MeU-5MeC-A-5MeC-5MeU-G-5MeU- 5MeU-G-5MeC-A-3¿ | Orphan Designation | - |
| cystic fibrosis | Antisense Oligonucleotide | Orphan Designation | - |